Parallel assessment of globin lentiviral transfer in induced pluripotent stem cells and adult hematopoietic stem cells derived from the same transplanted β-thalassemia patient

by Alisa Tubsuwan, Soumeya Abed, Annette Deichmann, Melanie D. Kardel, Cynthia Bartholomä, Alice Cheung, Olivier Negre, Zahra Kadri, Suthat Fucharoen, Christof Von Kalle, Emmanuel Payen, Stany Chrétien, Manfred Schmidt, Connie J. Eaves, Philippe Leboulch, Leïla Maouche-Chrétien
Published 2018

Betibeglogene autotemcel gene therapy for non–β⁰/β⁰ genotype β-thalassemia

by Franco Locatelli, Alexis A. Thompson, Janet L. Kwiatkowski, John B. Porter, Adrian J. Thrasher, Suradej Hongeng, Martin G. Sauer, Isabelle Thuret, Ashutosh Lal, Mattia Algeri, Jennifer Schneiderman, Timothy S. Olson, Ben Carpenter, Persis J. Amrolia, Usanarat Anurathapan, Axel Schambach, Christian Chabannon, Manfred Schmidt, Ivan Labik, Heidi Elliot, Ruiting Guo, Mohammed Asmal, Richard A. Colvin, Mark C. Walters
Published 2022